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Phase II clinical trial of neuroprotection with Phenytoin in acute optic neuritis

A case study of drug repurposing from Dr Raj Kapoor

neurons

13 January 2020

Principal Investigator: Dr Raj Kapoor, Institute of Neurology聽

Unmet Medical Need: Acute demyelinating optic neuritis, (common feature of multiple sclerosis) can damage vision through neurodegeneration in the optic nerve and in its fibres in the retina.聽

Current treatment: Corticosteroids help vision to recover more quickly, but do not improve on the overall extent of recovery compared to untreated.聽

Project:听

  • Inhibition of voltage-gated sodium channels is neuroprotective in preclinical models.聽聽
  • Is sodium-channel inhibition with phenytoin neuroprotective in patients with acute optic neuritis?
  • Ran randomised, placebo-controlled, double-blind phase 2 trial at two leading UK academic hospitals

Funders: National Multiple Sclerosis Society and the UK MS Society

Outcome: Trial results support the concept of neuroprotection using phenytoin to inhibit voltage-gated sodium channels in patients with acute optic neuritis聽

Next stage: Further investigation in larger clinical trials in optic neuritis and in relapsing multiple sclerosis is warranted.